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Neurogene Inc. (NGNE-8.60%) has submitted its annual report on Form 10-K filing for the fiscal year ended December 31, 2024.
The report details the company's progress in developing genetic medicines for rare neurological diseases, focusing on their proprietary transgene regulation technology, EXACTTM. The company's lead product candidate, NGN-401, is in a Phase 1/2 clinical trial for the treatment of Rett syndrome.
The clinical trial of NGN-401 is being conducted in the United States, the United Kingdom, and Australia. Interim data from the trial showed consistent improvements in clinical assessments and developmental milestones in participants receiving the 1E15 vg dose.
The company reported a serious adverse event in a participant receiving a higher dose of NGN-401, leading to the removal of the 3E15 vg dose from the trial protocol. Neurogene plans to continue enrolling participants at the 1E15 vg dose level.
Neurogene announced that NGN-401 has been selected for the FDA's START Pilot Program and has received RMAT designation, which may facilitate its development and regulatory review.
The company also disclosed financial results, including a net loss of $75.1 million for the year ended December 31, 2024, and an accumulated deficit of $262.3 million. Neurogene had cash, cash equivalents, and short-term investments totaling $312.4 million.
Neurogene highlighted its in-house manufacturing capabilities in Houston, Texas, which support the production of clinical-grade material for ongoing and future trials.
The company is evaluating options for its NGN-101 program for CLN5 Batten disease after the FDA denied RMAT designation, impacting the program's streamlined registrational pathway.
Neurogene's collaboration with the University of Edinburgh remains a key component of its research and development strategy, with an agreement extended through December 2026.
The report includes a summary of risks, such as the need for additional capital, reliance on third-party collaborations, and potential regulatory and market challenges.
This content was summarized by generative artificial intelligence using public filings retrieved from SEC.gov. The original data was derived from the Neurogene Inc. annual 10-K report dated March 24, 2025. To report an error, please email earnings@qz.com.